US: Food and Drug Administration has approved Bempedoic acid (NEXLETOL)—a once-daily, oral non-statin treatment for adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease (ASCVD), as an adjunct to diet and maximally tolerated statin therapy.
It is the first oral, once-daily, Non-Statin LDL-Cholesterol lowering medicine approved in the U.S. in nearly 20 Years for indicated patients. It lowers LDL-cholesterol with a first-in-class mechanism and fills an unmet need for affordable medicines for millions of patients with ASCVD or HeFH.Nexletol will be commercially available after March 2020.
The approval of NEXLETOL is supported by a global pivotal Phase 3 LDL-C lowering program conducted in more than 3,000 patients. In these studies, NEXLETOL provided an average of 18 per cent placebo corrected LDL-C lowering when used with moderate or high-intensity statins. Results from the Phase 3 development program have been published in The New England Journal of Medicine (040 Study), and The Journal of the American Medical Association (047 Study).
The FDA approval of NEXLETOL provides an important option for patients living with elevated LDL-C and ASCVD or increased risk for cardiovascular disease because of HeFH," said Christie M. Ballantyne, M.D., chairman of Esperion's Phase 3 Executive Committee and professor and chief of cardiology at Baylor College of Medicine in Houston. "There are millions of patients who are unable to reach their LDL-C targets despite available medicines. NEXLETOL is the first oral, once-daily, non-statin treatment option for indicated patients in nearly two decades."
NEXLETOL was generally well-tolerated in clinical studies. Label warnings and precautions include hyperuricemia, with the development of gout in a small percentage of patients, as well as increased risk of tendon rupture or injury. Overall in Phase 3 studies, the adverse events reported most frequently in patients who received NEXLETOL were generally reported at similar rates in patients who received placebo.
The most common adverse events reported with NEXLETOL (incidence ≥ 2% and greater than placebo) were upper respiratory tract infections, muscle spasms, hyperuricemia, back pain, abdominal pain or discomfort, bronchitis, pain in extremity, anemia, and elevated liver enzymes. The majority of adverse events reported with NEXLETOL were mild to moderate in severity and balanced in occurrence with adverse events in patients receiving placebo.